Why family needs $3.1 million to save daughter’s life

LIKE every parent, Meringandan mother Kellee Clarkson only wants what's best for her 16-month old daughter Wynter - and in this case it's a $3.1 million medical treatment.

After missing out on early testing, something only afforded to parents in NSW and ACT, Wynter was diagnosed with Spinal Muscular Atrophy type 1 at four months old.

Although young, the diagnosis came too late to prevent irreparable damage, the rare genetic disease causing loss of motor neurons, muscle wasting and a decreased life expectancy.

"I still feel guilt because if they had picked it up and treated her at birth, her life would have been normal," Mrs Clarkson said.

Wynter was placed on Spinraza, the only SMA treatment available in Australia. Producing the missing protein, it slows disease progression but can only be administered by lumbar puncture once every four months.

"Taking her to hospital and watching her go through the pain of a lumbar puncture is horrific," Mrs Clarkson said.

"She's too young to understand why it's happening, and we will have to do this for the rest of her life. She has PTSD from the visits, she's so scared of the pain."

Now the family is hanging its hopes on a $3.1 million wonder treatment, which has just completed its trial phase in America.

Zolgensma is a gene therapy which replaces the faulty gene in sufferers, allowing the production of protein vital to motor neuron development.

The treatment, which is administered in one dose, is not yet available in Australia. It will cost the family $3.1 million to import.

"You can't place a price on a child's life and from what we have seen and from what other families have said, this gene therapy is hands down the best," she said.

"The trial results have shown that 24 months after their dose children are reaching milestones - some are even walking.

"They also don't require ventilation to breathe, which is another reason we need the treatment. Wynter's respiratory system is weak and with SMA, bad respiratory function is typically why kids die"

Unfortunately, money isn't the only factor in this treatment - so is time.

"It can only be administered under the age of two, as the children become immune to the vector after that," she said.

"We have safely six months to raise the funds and administer the treatment.

"It's a huge ask for such an astronomical amount, but we are desperate for this treatment."

The couple has started up a GoFundMe page for baby Wynter, which has already raised $6000 over the past month.

Every bit will help the Clarkson family in securing Wynter's treatment. Click here to donate and share the cause with others.